Universität zu Köln

Niehaus, Ines Marina ORCID: 0000-0002-4214-6898 and Dintsios, Charalabos-Markos ORCID: 0000-0003-3469-092X (2018). Confirmatory versus explorative endpoint analysis: Decision-making on the basis of evidence available from market authorization and early benefit assessment for oncology drugs. Health Policy, 122 (6). S. 599 - 607. CLARE: ELSEVIER IRELAND LTD. ISSN 1872-6054

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Abstract

The early benefit assessment of pharmaceuticals in Germany and their preceding market authorization pursue different objectives. This is reflected by the inclusion of varying confirmatory endpoints within the evaluation of oncology drugs in early benefit assessment versus market authorization, with both relying on the same evidence. Data from assessments up to July 2015 are used to estimate the impact of explorative in comparison to confirmatory endpoints on market authorization and early benefit assessment by contrasting the benefit-risk ratio of EMA and the benefit-harm balance of the HTA jurisdiction. Agreement between market authorization and early benefit assessment is examined by Cohen's kappa (k). 21 of 41 assessments were considered in the analysis. Market authorization is more confirmatory than early benefit assessment because it includes a higher proportion of primary endpoints. The latter implies a primary endpoint to be relevant for the benefit-harm balance in only 67% of cases (0.078). Explorative mortality endpoints reached the highest agreement regarding the mutual consideration for the risk-benefit ratio and the benefit-harm balance (0.000). For explorative morbidity endpoints (-0.600), quality of life (-0.600) and side effects (-0.949) no agreement is ascertainable. To warrant a broader confirmatory basis for decisions supported by HTA, closer inter-institutional cooperation of approval authorities and HTA jurisdictions by means of reliable joint advice for manufacturers regarding endpoint definition would be favorable. (C) 2018 Elsevier B.V. All rights reserved.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Niehaus, Ines Marina UNSPECIFIED orcid.org/0000-0002-4214-6898 UNSPECIFIED Dintsios, Charalabos-Markos UNSPECIFIED orcid.org/0000-0003-3469-092X UNSPECIFIED
URN:	urn:nbn:de:hbz:38-184189
DOI:	10.1016/j.healthpol.2018.03.017
Journal or Publication Title:	Health Policy
Volume:	122
Number:	6
Page Range:	S. 599 - 607
Date:	2018
Publisher:	ELSEVIER IRELAND LTD
Place of Publication:	CLARE
ISSN:	1872-6054
Language:	English
Faculty:	Faculty of Medicine
Divisions:	Faculty of Medicine > Sonstiges > Zentrum für Versorgungsforschung Köln
Subjects:	Medical sciences Medicine
Uncontrolled Keywords:	Keywords Language GERMANY; AGREEMENT; KAPPA Multiple languages Health Care Sciences & Services; Health Policy & Services Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/18418